Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene
Dolan Sondhi, Emma C. Scott, Alvin Chen, Neil R. Hackett, Andrew M.S. Wong, Agnieszka Kubiak, Hemanth R. Nelvagal, Yewande Pearse, Susan L. Cotman, Jonathan D. Cooper, Ronald G. Crystal
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